FDA and NIH grant clearance for VistaGen to initiate AV-101 Phase II MDD trial

AV-101 is a clinical-stage prodrug candidate that readily gains access to the central nervous system (CNS) after systemic administration and is rapidly converted in vivo to its active metabolite, 7-chlorokynurenic acid (7-Cl-KYNA).

7-Cl-KYNA is a well-characterized, potent and highly-selective antagonist of the glycine-binding co-agonist (GlyB) site of the N-methyl-D-aspartate receptor (NMDAR).

The US National Institutes of Mental Health (NIMH) Neurobiology and Treatment of Mood Disorders chief and Experimental Therapeutics and Pathophysiology Branch chief Dr Carlos Zarate will be the principal investigator of the trial.

VistaGen Therapeutics president and chief scientific officer Ralph Snodgrass said: "The NIMH and several key leaders in the field with clinical experience using ketamine to treat MDD provided valuable expert advice on the design of our Phase II MDD study.

"We are grateful for their assistance. With their help, we have now achieved an important regulatory milestone for our AV-101 clinical development program.

"The pharmacology and existing clinical safety data and preclinical efficacy data point to AV-101’s potential to provide a transformative advancement in the treatment of MDD, in a manner fundamentally different from all currently approved antidepressants."

The randomized, double-blind, placebo-controlled, crossover Phase II trial, which will be conducted at the NIMH, will include around 25 patients with MDD.

The NIH-funded trial will evaluate the efficacy and safety of a single oral dose of AV-101 administered once per day for 14 days.

Patient enrollment is expected to be initiated by the NIMH and VistaGen in the third quarter of this year.